Scientific Conference
March 19-22, 2017 • Arlington, VA
Advancing
Scientific
Breakthroughs
To Save and
Improve Lives
mda.org
#MDASciCon
Muscular Dystrophy Association
Conference Information
Disclosure Statement
It is MDA’s policy to ensure objectivity and scientific
rigor in all of its educational activities. All participating
speakers are required to disclose at the beginning of
their presentation any financial relationships related to
the subject matter to be discussed. Any such financial
relationships of spouse/partner should also be disclosed.
Poster Sessions
Poster judging for trainees takes place on Monday, March
20 (6:45-8:45 p.m.). All trainees should be by their posters
during this time. Awardees will be notified via email no later
than Tuesday morning, March 21. Poster awards will be
announced to the general audience on Wednesday, March
20, 8:30-9 a.m. Poster awardees will each give a threeminute oral presentation during this time, using a maximum
of two slides.
Non-trainees should stand by their posters as follows:
Odd numbers: Monday, March 20 (6:45-8:45 p.m.)
Even numbers: Tuesday, March 21 (6:30-8:30 p.m.).
Please make sure to remove your poster from the exhibit
hall by 8 a.m. on Wednesday, March 22. MDA will not be
responsible for any posters remaining after this time.
Wireless Login Instructions:
1.
Please select Wi-Fi Internet Access:
ACCELERON
2. Once ACCELERON is selected, it will route you to the
HYATT splash page and will request your access code.
3. Please enter: mda2017 (this code allows you to
access Wi-Fi in all meeting spaces).
4. Press enter.
5. You will see the Hyatt Crystal City home page, which
indicates that you have gained access to the Wi-Fi.
2
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Advancing Scientific Breakthroughs
To Save and Improve Lives
Welcome to the 2017 MDA Scientific Conference
R. Rodney Howell, M.D.
Chairman
MDA Board of Directors
Thank you for joining us at the 2017 MDA Scientific Conference. We’re excited to have
you with us as we work collectively toward advancing scientific breakthroughs to save
and improve lives.
We’re gathering at a special time in the history of MDA and the neuromuscular disease
field, as we’ve recently seen three drugs, all developed and tested with critical support
from MDA, be granted FDA approval to treat diseases in our program. Three drugs in six
months!
Following these successes, our sense of urgency to find treatments for all the diseases
under our umbrella has only been heightened, our efforts redoubled. We are certain
that these approvals will provide inspiration and motivation for academics, biotechs,
pharmaceutical companies and other key industry stakeholders to galvanize their
efforts and spur advances in drug development across the board. Perhaps even more
importantly, these new treatment options have brought an even greater sense of hope to
the individuals and families we serve, those who are counting on us — on all of us — to
drive the development of therapies and cures.
Your participation over the next three days is proof of your commitment to working
together toward our common goals, not only to keep up this newfound momentum but
to accelerate progress toward the solutions so many children and adults are waiting for.
Our 2017 conference is focused on increasing knowledge of disease causes, identifying
new therapeutic targets and innovative technologies, and discussing new advances
in preclinical and clinical research — all aimed at accelerating drug development and
targeted treatments for neuromuscular diseases. We plan to cover a broad range of
topics, with ample opportunity for dialogue and exchange of ideas.
Because of you and the work you are doing throughout the United States and around the
world, individuals with neuromuscular diseases are living longer and growing stronger.
We truly appreciate your passion and partnership as we work together for strength,
independence and life. Thank you for all you do every day to help accelerate progress in
our fight to free individuals — and the families who love them — from the harmful effects
of neuromuscular diseases.
3
Keynote Speakers
Matthew Porteus, M.D., Ph.D.
Stanford University
Genome Editing for Genetic Diseases: Challenges and Opportunities
Matthew Porteus is an associate professor at Stanford. His research program has made important
discoveries in advancing the field of genome editing, including the first use of genome editing using
engineered nucleases in human cells and optimizing the use of the CRISPR/Cas9 system in primary
human stem cells. As an attending physician, Porteus cares for children undergoing bone marrow
transplantation. His goal is to combine his research and clinical interests to bring innovative curative
therapies to patients.
Mark Schnitzer, Ph.D.
Stanford University
In Vivo Imaging of Human Sarcomere Twitch Dynamics in Individual Motor Units
Mark Schnitzer is an associate professor at Stanford University and an investigator of the Howard
Hughes Medical Institute. His research concerns the innovation of novel optical imaging technologies,
and their use in the pursuit of understanding neural circuits and muscle microstructure and contractile
dynamics. His approaches are being used clinically to better visualize how muscle structure and
function is compromised in the context of neuromuscular diseases.
Enabling Technology Speaker
Sandeep Robert Datta, M.D., Ph.D.
Harvard Medical School
Deep Phenotyping Using Motion Sequencing
Sandeep Robert Datta is an assistant professor at Harvard Medical School. Datta’s lab focuses on
understanding how the brain transforms information about sensory cues into patterns of motivated
action. This work involves studying genes involved in detecting odors, revealing patterns of neural
activity that encode sensory maps of the outside world, and probing the fundamental statistical
structure of behavior itself. His innovative approach for studying the modular structure of behavior can
be used as a sensitive read-out of the effects of disease mutations and potential therapeutics.
4
Advancing Scientific Breakthroughs
To Save and Improve Lives
Sunday, March 19, 2017
3 – 6:30 p.m. Registration. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Regency Foyer
Keynote Speakers . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Regency CD Ctr EF
5 – 6:30 p.m. Genome Editing for Genetic Diseases: Challenges and Opportunities
Matthew Porteus, M.D., Ph.D., Stanford University
In Vivo Imaging of Human Sarcomere Twitch Dynamics in Individual Motor Units
Mark Schnitzer, Ph.D., Stanford University
6:30 – 8:30 p.m. Champions Reception . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Independence Center A
Monday, March 20, 2017
6 – 6:45 a.m. MDA Team Momentum Morning Fun Run (optional) . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Lobby
7 – 8 a.m. Breakfast . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Independence Center B
Opening Session . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Regency CD Ctr EF
8 – 8:30 a.m. Welcome and Opening Remarks
Session 1: Genetics/Epigenetics/Gene Discovery . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Regency CD Ctr EF
Session Chairs: Robert Brown and Monkol Lek
8:30 – 9 a.m. Systematic Identification of Causal Mutations in Severe Muscle Diseases Using
Various Genomic Technologies
Monkol Lek, Ph.D., Massachusetts General Hospital
9 – 9:30 a.m. Collagen VI-Related Dystrophy — Mutation-Specific Approaches to Dominant Mutations
Carsten Bönnemann, M.D., National Institutes of Health
9:30 – 9:45 a.m. The Role of Harakiri, a Mitochondrial Apoptosis Mediator, in Maintaining Membrane
Stability of Myositis Muscle
Jessica Boehler, Children’s National Health System
9:45 - 10 a.m. Epigenetic Silencing in Friedreich's Ataxia is Caused by Hypermethylation of the FXN CpG Island Shore
Sanjay Bidichandani, MBBS, Ph.D., University of Oklahoma Health Sciences Center
10 – 10:30 a.m. Coffee Break . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Independence Center A
10:30 – 11 a.m. TBD
Robert Brown, M.D., Ph.D., University of Massachusetts Medical School
11 – 11:15 a.m. Targeting the Long Non-Coding RNA SMN-AS1 as a Treatment for Spinal Muscular Atrophy
Constantin d’Ydewalle, Ph.D., Johns Hopkins School of Medicine
11:15 – 11:30 a.m. Mutations in INPP5K Cause a Novel Syndrome with Congenital Muscular Dystrophy,
Intellectual Disability and Cataracts
M. Chiara Manzini, Ph.D., George Washington University
5
Monday, March 20, 2017
11:30 a.m.– 12 p.m. Facioscapulohumeral Muscular Dystrophy is a Model Epigenetic Disease
Peter Jones, Ph.D., University of Nevada
12 – 1:30 p.m. Lunch . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Independence Center B
Enabling Technology . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Regency CD Ctr EF
1:30 – 2 p.m. Deep Phenotyping Using Motion Sequencing
Sandeep Robert Datta, M.D., Ph.D., Harvard Medical School
Session 2: Precision Medicine Approaches . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Regency CD Ctr EF
Session Chairs: Adrian Krainer and Matthew Wood
2 – 2:30 p.m. Targeted Antisense Therapy for Spinal Muscular Atrophy
Adrian Krainer, Ph.D., Cold Spring Harbor Laboratory
2:30 – 2:45 p.m. Translating DUX4-Targeted RNAi Therapy for Facioscapulohumeral Muscular Dystrophy
Lindsay Wallace, Ph.D., Nationwide Children’s Hospital
2:45 – 3 p.m. Dystrophin Induction Following Muscle-Specific AAV-CRISPR/Cas9-Mediated Editing of
the Murine Dystrophin Gene
Jeffrey Chamberlain, Ph.D., University of Washington
3 – 3:30 p.m. Coffee Break . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Independence A
3:30 – 4 p.m. Advanced Oligonucleotide Therapeutics for Neuromuscular Disease
Matthew Wood, Ph.D., University of Oxford
4 – 4:30 p.m. IMPEDE: Inhibition of Microsatellite Promoted Expression of Deleterious Expansions in
Myotonic Dystrophy
Andrew Berglund, Ph.D., University of Florida
4:30 - 5 p.m. Free Time
Session 3A: Channelopathies
Regency C & D
Session 3B: Nuclear Membranes
Washington Room
Session 3C: Mitochondrial
Myopathies Regency E & F
Session Chairs: Steve Cannon
and Mark Rich
Session Chairs: Christopher Donnelly
and Lori Wallrath
Session Chair: Giovanni Manfredi
5 – 5:30 p.m.
5 – 5:15 p.m.
5 – 5:30 p.m.
New Insights on Exercise-Triggered
Weakness in Hypokalemic Periodic
Paralysis
FG Nucleoporin Dysfunction in
Amyotrophic Lateral Sclerosis (ALS)
Glutamate Anaplerosis as a
Mechanism of Metabolic Adaptation in
Mitochondrial Diseases
Steve Cannon, M.D., Ph.D.,
University of California,
Los Angeles
6
Christopher Donnelly, Ph.D.,
University of Pittsburgh
Giovanni Manfredi, M.D., Ph.D.,
Weill Medical College
Advancing Scientific Breakthroughs
To Save and Improve Lives
Monday, March 20, 2017
5:30 – 6 p.m.
5:15 – 5:30 p.m.
5:30 – 5:45 p.m.
Early Cl− and K+ Channel Defects in
Huntington’s Disease Skeletal Muscle
Reveal Disrupted Maturation
Disruption of Nucleocytoplasmic
and Chemokine Signaling and
Proteostasis by Loss of the
Nucleoporin, Ranbp2, in Motoneurons
Causes Amyotrophic Lateral Sclerosis
(ALS)-Like Syndromes
Investigation of a Mouse Model for
CHCHD10 Mitochondrial Myopathy
Paulo Ferreira, Ph.D.,
Duke University Medical Center
Regulation of Mitochondrial Complex
I Biogenesis in Drosophila Skeletal
Muscles
Andrew Voss, Ph.D.,
Wright State University
6 – 6:30 p.m.
Discovery of a Persistent Inward
Sodium Current in Skeletal Muscle
That Contributes to Myotonic Action
Potentials
Mark Rich, M.D., Ph.D.,
Wright State University
5:30 – 5:45 p.m.
uclear Envelope Mutations
N
Associated with Emery-Dreifuss
Muscular Dystrophy Cause Softer and
More Fragile Nuclei
Gregory Fedorchak,
Cornell University
5:45 – 6 p.m.
egulation of Nucleus-Nucleus
R
Interactions During Myonuclear
Movement
Teepu Siddique, M.D.,
Northwestern University
5:45 – 6 p.m.
Edward Owusu-Ansah, Ph.D.,
Columbia University
6 – 6:30 p.m.
Development of a Pharmacological
Therapy for Thymidine Kinase 2 (TK2)
Deficiency
Michio Hirano, M.D.,
Columbia University Medical Center
Eric Folker, Ph.D.,
Boston College
6 – 6:15 p.m.
Rescue of Emerin-Null Myogenic
Progenitor Differentiation by
Inhibition of Multiple MAP Kinases or
Activation of HDAC3
Joseph Ellis,
University of the Sciences
6:15 – 6:30 p.m.
Loss of Proteostasis and Redox
Homeostasis in Nuclear Envelope
Muscular Dystrophy
Lori Wallrath, Ph.D.,
University of Iowa
6:45 – 8:45 p.m. Poster Session and Reception. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Independence Center A
Poster Judging - Trainees
Posters - Non-Trainees (Odd numbers)
— Dinner On Your Own —
7
Tuesday, March 21, 2017
7 – 8 a.m. Breakfast . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Independence Center B
Session 4A: Dystrophin Glycoprotein
Complex Diseases
Regency C & D
Session 4B: Motor and Peripheral
Neuron Diseases
Regency E & F
Session Chairs: Aaron Beedle and Elizabeth McNally
Session Chairs: Tania Gendron and Angelo Lepore
8 – 8:30 a.m.
8 – 8:30 a.m.
Evidence of mTORC1 Activity in the Pathogenesis of
Dystroglycanopathy-Type Muscular Dystrophy
Poly(GP) Dipeptide Repeat Proteins as a Pharmacodynamics
Biomarker for the Development of C9ALS Therapies
Aaron Beedle, Ph.D., Binghamton University
Tania Gendron, Ph.D., Mayo Clinic - Jacksonville
8:30 – 8:45 a.m.
8:30 – 9 a.m.
Intracellular Signaling Responses that Mediate Membrane
Repair in Striated Muscle Can Compensate for Membrane
Fragility in Muscular Dystrophy
MFN1 Augmentation to Suppress Toxicity in a Novel
Mouse Model of CMT2A
Noah Weisleder, Ph.D., Ohio State University
8:45 – 9 a.m.
Vamorolone Improves Inflammation Via the GR in Addition
to Heart Health Via the MR in mdx Mice
Christopher Heier, Ph.D.,
Children’s National Medical Center
Robert Baloh, M.D., Ph.D., Cedars-Sinai Medical Center
9 – 9:30 a.m.
Dissecting DNA Methylation Dynamics During the
Development and Function of Human Motor Neurons
Evangelos Kiskinis, Ph.D., Northwestern University
9 – 9:30 a.m.
Molecular Regulation of Muscle Stem Cell Asymmetric
Division
Michael Rudnicki, Ph.D.,
Ottawa Hospital Research Institute
8
9:30 – 10 a.m. Coffee Break . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Independence A
Session 4A: Dystrophin Glycoprotein
Complex Diseases
Regency C & D
Session 4B: Motor and Peripheral
Neuron Diseases
Regency E & F
10 – 10:30 a.m.
10 – 10:30 a.m.
Modifiers of Muscular Dystrophy
Nuclear Transport and Pores in Neurodegenerative Diseases
Elizabeth McNally, M.D., Ph.D., Northwestern University
Jeffrey Rothstein, M.D., Ph.D., Johns Hopkins University
Advancing Scientific Breakthroughs
To Save and Improve Lives
Tuesday, March 21, 2017
10:30 – 11 a.m.
10:30 – 10:45 a.m.
Role of Osteopontin in Promotion of DMD Pathogenesis
TMEM184b Controls Axon Degeneration and
Neuromuscular Junction Structure and Modulates
Autophagy
Melissa Spencer, Ph.D.,
University of California - Los Angeles
11 – 11:15 a.m.
Exon 51 Skipping Mediated by WVE-210201, a Potential
Disease-Modifying Therapy for Duchenne Muscular Dystrophy
Martha Bhattacharya, Ph.D., University of Arizona
10:45 – 11 a.m.
Kirsten Gruis, M.D., WAVE Life Sciences
Development of a Pharmacologically-Induced Intermediate
Mouse Model to Identify Protective Genetic Modifiers of
Spinal Muscular Atrophy
11:15 – 11:30 a.m.
Kevin Kaifer, University of Missouri
miR-146a Inhibits Dystrophin Production and Promotes
Inflammation in Multiple Muscle Disorders
11 – 11:30 a.m.
Alyson Fiorillo, Ph.D.,
Children’s National Medical Center
Toward Therapeutic Intervention in ALS: Role of Ephrin
Signaling in Astrocytes
Angelo Lepore, Ph.D., Thomas Jefferson University
11:30 a.m. – 1 p.m. Lunch . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Independence Center B
Session 5A: Repeat Expansion Diseases
Regency C & D
Session 5B: Protein Misfolding and Turnover
Regency E & F
Session Chairs: Laura Ranum and Eric Wang
Session Chairs: Neil Cashman and Rudolph Kley
1 – 1:30 p.m.
1 – 1:30 p.m.
Regulation of RNA Processing in Myotonic Dystrophy
Initiation, Propagation and Inhibition of SOD1 Misfolding in
Amyotrophic Lateral Sclerosis
Eric Wang, Ph.D., University of Florida
1:30 – 2 p.m.
Facioscapulohumeral Dystrophy: Molecular Mechanisms
and Therapeutic Opportunities
Stephen Tapscott, M.D., Ph.D.,
Fred Hutchinson Cancer Research Center
2 – 2:30 p.m.
Inducible and Reversible Phenotypes in a Novel Mouse
Model of Friedreich’s Ataxia
Vijayendran Chandran, Ph.D., University of Florida
Neil Cashman, M.D., University of British Columbia
1:30 – 2 p.m.
Functional Analysis of the PABPN1 Protein Provides
Insight into the Mechanisms Underlying Oculopharyngeal
Muscular Dystrophy
Anita Corbett, Ph.D., Emory University
2 – 2:30 p.m.
Defining the Role of Skeletal Muscle in X-Linked Spinal
and Bulbar Muscular Atrophy: Implications for Therapy
Development in Motor Neuron Disease
Albert LaSpada, M.D., Ph.D.,
University of California, San Diego
2:30 – 3 p.m. Coffee Break . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Independence A
9
Tuesday, March 21, 2017
Session 5A: Repeat Expansion Diseases
Regency C & D
Session 5B: Protein Misfolding and Turnover
Regency E & F
3 – 3:30 p.m.
3 – 3:30 p.m.
RAN Translation Regulated by Muscleblind Proteins in
Myotonic Dystrophy Type 2
Regulation of the Ubiquitin-Proteasome Pathway in Normal
and Atrophying Muscles and with Exercise
Laura Ranum, Ph.D., University of Florida
Alfred Goldberg, Ph.D., Harvard Medical School
3:30 – 4 p.m.
3:30 – 4 p.m.
Building a Biomarker for Myotonic Dystrophy Type 1 (DM1)
Protein Aggregation in Myofibrillar Myopathies
Charles Thornton, M.D.,
University of Rochester Medical Center
Rudolf Kley, M.D., Ruhr-Universität Bochum
4 – 4:30 p.m Free Time
Workshop 1A: Scientific Careers Outside
of Academia
Regency C & D
Workshop 1B: How to Move Forward with
your Drug Idea: Strategies and Resources
for Academics Regency E & F
Moderator: Amanda Haidet-Phillips
Moderators: Laura Hagerty and Lianna Orlando
4:30 – 6 p.m.
4:30 – 6 p.m.
Panelists:
Panelists:
Adam Berger, Ph.D., U.S. Food and Drug Administration
Frank Bennett, Ph.D., Ionis Pharmaceuticals
Thomas Cheever, Ph.D., National Institutes of Health
Michio Hirano, M.D., Columbia University Medical Center
Grace Ha, Ph.D., Deloitte Consulting
Eric Hoffman, Ph.D., Binghamton University
Kimberly Long, Ph.D., Scholar Rock
Dione Kobayashi, Ph.D., Cydan Development
Valerie Thompson, Ph.D., Science Magazine
Jane Muir, RTTP, University of Florida
6:30 – 8:30 p.m. Poster Session and Reception. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Independence Center A
Posters - Non-Trainees (Even Numbers)
— Dinner On Your Own —
10
Advancing Scientific Breakthroughs
To Save and Improve Lives
Wednesday, March 22, 2017
7 – 8 a.m. Breakfast . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Independence Center B
Opening Session . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Regency CD Ctr EF
8 – 8:30 am Advocacy and its Importance on Advancing Neuromuscular Disease Research
Kristin Stephenson, M.H.A, J.D.
8:30 – 9 a.m. Poster Award Announcements and Trainee Presentations
Session 6: Clinical Trials . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Regency CD Ctr EF
Session Chairs: Laura Hagerty and Lianna Orlando
9 – 9:30 a.m. A Novel Therapeutic Approach in Spinal Muscular Atrophy (SMA): The Clinical
Development of Nusinersen
Wildon Farwell, M.D., Biogen
9:30 – 9:45 a.m. Phase 1/2 Clinical Trial of Clenbuterol in Pompe Disease Patients Stably Treated with ERT
Edward Smith, M.D., Duke University Hospital
9:45 – 10 a.m. A Phase 1b/2a Trial of Ibudilast (MN-166-ALS-1201), a Phosphodiesterase Type 4
Inhibitor for Amyotrophic Lateral Sclerosis (ALS)
Benjamin Brooks, M.D., Carolinas Health Care System Neurosciences Institute
10 – 10:30 a.m. Coffee Break . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Regency E & F Foyer
10:30 – 10:45 a.m. Trehalose Treatment For Oculopharyngeal Muscular Dystrophy (OPMD): Safety,
Pharmacokinetics and Efficacy Signals in an Open Label Phase 2 Trial
Zohar Argov, M.D., Hadassah-Hebrew University Medical School
10:45 – 11 a.m. Effects of Long-Term Treatment With Eteplirsen on Cardiac Function: Left Ventricular
Ejection Fraction in Eteplirsen-Treated Patients vs Disease Natural History
Petra Duda, M.D., Ph.D., Sarepta Therapeutics
11 – 11:15 a.m. Preliminary Pharmacokinetic and Safety Data in Patients With Pompe Disease in
First-In-Human Study Receiving ATB200/AT2221
Swati Sathe, M.D., Amicus Therapeutics
11:15 – 11:45 a.m. MoveDMD: Phase 1/2 Trial of Edasalonexent, an NF-κB Inhibitor, in 4- to 7-Year-Old
Patients with Duchenne Muscular Dystrophy
Joanne Donovan, M.D., Ph.D., Catabasis Pharmaceuticals
— Conference Concludes —
11
THIRD FLOOR
RESTROOMS
PRESIDENT’S
QUARTERS
ROOSEVELT ROOM
ARLINGTON ROOM
ELEVATORS
LINCOLN ROOM
PRINCE WILLIAM ROOM
JEFFERSON ROOM
KENNEDY ROOM
PLAN
ors
ATRIUM
Hotel Map:
THIRD FLOOR
HYATT STAYFIT GYM
POOL
Third Floor
WHIRLPOOL
RESTROOMS
PRESIDENT’S
QUARTERS
ROOSEVELT ROOM
ARLINGTON ROOM
ELEVATORS
LINCOLN ROOM
VIRGINIA
ROOM
FAIRFAX ROOM
PRINCE WILLIAM ROOM
JEFFERSON ROOM
KENNEDY ROOM
SECOND FLOOR
VIRGINIA
ROOM
FAIRFAX ROOM
TIDEWATER I
ATRIUM
TIDEWATER II
ELEVATORS
BOARDROOM
HYATT STAYFIT GYM
ANTEROOM
CINNABAR
RESTAURANT
ROOFTOP
POOL
ATRIUM
WHIRLPOOL
TERRACE
RESTROOMS
Independence Level
TIDEWATER I
SECOND FLOOR
TIDEWATER II
ELEVATORS
INDEPENDENCE LEVEL
BOARDROOM
BAR
ANTEROOM
CINNABAR
RESTAURANT
ATRIUM
ROOFTOP
INDEPENDENCE
CENTER B
TERRACE
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ELEVATORS
INDEPENDENCE
CENTER A
BAR
ATRIUM
INDEPENDENCE
FOYER
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INDEPENDENCE
CAPITOL
CENTER BROOM
Ballroom Level
BUSINESS
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INDEPENDENCE
OFFICE
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INDEPENDENCE
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CONVENTION
OFFICE
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REGENCY
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CENTER
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CONVENTION
OFFICE
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CAPITOL
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POTOMAC
ROOM
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ATRIUM
CENTER
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B
WASHINGTON
ROOM
POTOMAC
ROOM
CONFERENCE
THEATER
Advancing Scientific Breakthroughs
To Save and Improve Lives
2017 Scientific Conference Sponsors
The Muscular Dystrophy Association is grateful to the following companies for sponsoring the 2017 Scientific Conference.
Together with our sponsors, MDA is advancing the scientific breakthroughs that will save and improve lives.
Hope Sponsors:
Platinum Sponsors:
Poster Session Sponsor:
Poster Session and Reception Sponsor:
Wi-Fi and Charging Station Sponsor:
Notepad Sponsor:
Pen Sponsor
Circle of Strength Sponsors:
Champions Reception Sponsor:
Coffee Break Sponsors:
In-Kind Supporters:
Hyatt Regency - Crystal City
Marquardt Printing
PSAV
Shepard Exposition Services
Single Digits
13
DG
MDA
Research
Grants
MVP
MDA Venture Philanthropy
MDA Venture Philanthropy is the
Muscular Dystrophy Association’s
drug development program,
exclusively focused on funding the
discovery and clinical application
of treatments and cures for
neuromuscular diseases.
Who can apply?
Biotechnology, pharmaceutical or
other companies are eligible for
MVP funding. MVP also may choose
to invest in academic projects
with a clear path toward clinical
development.
What kinds of projects are funded?
MVP funds projects from proofof-principle studies through
phase 2 trials. Projects may
include optimization, toxicology,
manufacturing and scale-up, preIND, phase 1 or early phase 2 trials.
For-profit entities are required to
contribute funds that match or
exceed MDA’s contribution. MDA
will negotiate return on investment.
Projects are milestone-driven, with
funds released according to the
completion of pre-agreed-upon
sections of the project.
What kinds of projects are funded?
Development Grant
Development Grants are awarded to
researchers on the brink of becoming
independent investigators, and are
intended as seed money to help launch
the scientific programs of promising new
neuromuscular disease researchers.
Development grants total $60,000 per
year, for one to three years.
Who can apply?
The applicant should hold a Ph.D.,
M.D. or other advanced degree, and
must be a member of a research
team operating under the guidance
of a principal investigator working
on one of the diseases in MDA’s
program. Applicants must have a
strong record of achievement with
their postdoctoral mentor and be at
least 18 months, but no more than
60 months, from receiving their most
advanced degree (Ph.D. or M.D.).
What kinds of projects are funded?
Projects should be hypothesis-driven
and used to investigate disease
mechanism, potential therapeutic
targets, animal models, drug screens,
etc., for a disease in MDA’s program.
RG
Any research related to therapy
development for the diseases covered
by MDA may be funded through
this mechanism. Projects should
be hypothesis-driven and used for
investigating disease mechanism,
potential therapeutic targets, animal
models, drug screens, etc.
HCTG
Human Clinical Trial Grant
Human Clinical Trial Grants are
designed to support both clinical
trials of compounds already on the
market and clinical studies performed
at academic medical centers.
(Phase 1 and phase 2a trials of novel
therapeutics should be submitted as
MDA Venture Philanthropy projects.)
HCTG grants may last up to three
years and are milestone-driven,
with funds released according to
the completion of pre-agreed-upon
sections of the project.
Who can apply?
The applicant should hold a Ph.D.,
M.D. or other equivalent advanced
degree and be an independent
investigator.
What kinds of projects are funded?
Research Grant
Research Grants are awarded to
established investigators. These
grants total $100,000 per year, for
one to three years. In a very limited
number of cases, awards will exceed
$100,000/year but pre-approval is
required before submission.
Human Clinical Trial Grants support
clinical trials of repurposed drugs,
non-drug interventions, natural history
studies and clinical studies performed
at academic medical centers. All trials
and studies must be of relevance to
MDA-covered diseases, FDA-approved
(if appropriate), and approved through
an Institutional Review Board.
Who can apply?
Independent investigators from
academic or corporate research
institutions are eligible to apply.
Muscular Dystrophy Association • mda.org/researchgrants
14
RIG
Research Infrastructure Grant
Research Infrastructure Grants fund
the development of infrastructure —
tools, techniques or services — that
will be of use to the neuromuscular
disease research community for the
purpose of therapy development.
Who can apply?
Independent investigators from
academic, non-profit, or other research
institutions may apply.
What kinds of projects are funded?
Any research designed to support the
development of tools, techniques and
services of need to the neuromuscular
research community may be funded
through this grant type. Recipients of an
MDA Infrastructure Grant must develop a
plan to make this MDA-funded resource
available to the research community,
and have a plan for sustaining the
infrastructure beyond the period of MDA
support. Letters of support from the
community are an essential part of the
application.
CRTS
Clinical Research
Training Scholarship
MDA has partnered with the American
Academy of Neurology and the
American Brain Foundation to offer
training scholarships to early-stage
investigators entering the field of
academic clinical neuromuscular
research. Awards total $55,000 per
year for two years, plus a $10,000-peryear stipend to support education and
research-related costs.
Who can apply?
Applicants must have an M.D. and
must have completed residency within
the past five years of the start date of
the award (July 1).
What kinds of projects are funded?
Projects should relate to patientoriented research or translational
research designed to develop
treatments or enhance diagnosis
for diseases covered under the MDA
umbrella.
CG
Conference Grant
MDA supports conferences, meetings,
and workshops that facilitate the
exchange of scientific ideas and crucial
information relevant to diseases in
MDA’s program. Awards total up to
$10,000.
Who can apply?
Independent investigators from
academic, nonprofit, or other research
institutions may apply.
What kinds of projects are funded?
Conferences should relate to diseases
covered in MDA’s program and
can include research conferences,
symposiums focused on new
techniques or therapeutic strategies,
workshops to update clinical care
guidelines, or meetings that promote
collaboration across diseases.
Grant Cycle Information: RG, DG
Spring Review Cycle
Letter of
Intent Due
Dec. 15
Grant
Application
Due
Funding
Decisions
Funding
Start Date
Grant
Application
Due
Funding
Decisions
Funding
Start Date
Jan. 31
May/June
Aug. 1
Fall Review Cycle
Letter of
Intent Due
June 15
July 15
Nov./Dec.
Feb. 1
MVP Letter of Intent due dates are: March 1, June 1, Sept. 1, Dec. 1.
HCTG, RIG and CG operate on rolling deadlines.
CRTS applications are due Oct. 1. Visit www.AAN.com/view/ResearchProgram to apply for these awards.
For general inquiries, please contact [email protected].
Muscular Dystrophy Association • mda.org/researchgrants
15
MDA is leading the fight to free individuals — and the families
who love them — from the harm of muscular dystrophy, ALS
and related muscle-debilitating diseases that take away
physical strength, independence and life. We use our collective
strength to help kids and adults live longer and grow stronger
by finding research breakthroughs across diseases; caring
for individuals from day one; and empowering families with
services and support in hometowns across America.
Joe Akmakjian
2017 MDA National
Ambassador
mda.org
#MDASciCon
Muscular Dystrophy Association